FDA has approved Rocket Pharmaceuticals’ gene therapy Kresladi (marne-cel) for leukocyte adhesion deficiency type I, and the therapy has earned a rare pediatric disease priority review voucher (PRV). The company said Kresladi is the first BLA successfully to pass CBER this year, with commercial launch planned by year-end. Rocket’s one-and-done approach delivers an engineered genetic payload intended to restore MAC-1 function in patients with LADI-I, a rare, life-threatening immunodeficiency. The approval adds another entry to a still-narrow set of gene therapies reaching late-stage regulatory clearance. Investors and clinicians will now watch post-launch demand, evidence generation in real-world use, and any follow-on label expansion as the PRV’s downstream value remains an added strategic lever for the company.